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NCT06382155 | RECRUITING | Idiopathic Short Stature

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
Sponsor:

BioMarin Pharmaceutical

Brief Summary:

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH), in children with idiopathic short stature (ISS).

Condition or disease

Idiopathic Short Stature

Intervention/treatment

Vosoritide Injection

Human Growth Hormone

Placebo

Phase

PHASE2

Detailed Description:

Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.

Study Type : INTERVENTIONAL
Estimated Enrollment : 100 participants
Masking : QUADRUPLE
Primary Purpose : TREATMENT
Official Title : A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
Actual Study Start Date : 2024-10-21
Estimated Primary Completion Date : 2026-06
Estimated Study Completion Date : 2036-12

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 3 Years to 10 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Key Inclusion Criteria
  • 1. Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts
  • 2. Tanner Stage 1, at time of signing the ICF (unless too young to stage).
  • Key Exclusions
    • 1. Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome)
    • 2. Previous treatment with a growth promoting agent
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Location Details

NCT06382155

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, Georgia

Centricity Research

Columbus, Georgia, United States, 31904

RECRUITING

United States, Idaho

St. Luke's Children's Endocrinology

Boise, Idaho, United States, 83712